FDA Workshop: Leveraging Randomized Clinical Trials to Generate Real-World Evidence for Regulatory Purposes

Dear All:

The idea of using real-world evidence (RWE) as a way to support development of novel agents (including antibiotics!) has always intrigued. As you may recall, the 21st Century Cures Act (Cures Act) gave FDA a mandate to evaluate the potential use of RWE to support drug approval.

There was a workshop on this on 13 Sep 2017 (link) and now we have the announcement of a 2nd workshop on 11-12 July 2019 (link) to discuss how randomized clinical trial designs can use real-world data (RWD) to generate RWE. Quoting directly from the announcement:

• “This workshop will explore key considerations for using randomized clinical trial designs and RWD to generate RWE, particularly in clinical care settings.
• “Considerations for discussion include: (1) Selection of interventions appropriate in clinical care settings, (2) study design elements and study populations, (3) capturing outcomes in clinical care settings, and (4) addressing potential challenges around blinding, randomization, and bias.
• “The workshop will also explore regulatory considerations for randomized clinical trials using RWD, such as safety and product monitoring and maintaining data integrity.”

Intriguing, no? Frustratingly, this workshop overlaps in part with the 12 July LPAD workshop (link), but that’s just how it goes … the announcement does indicate that video from the meeting will subsequently be available. 

All best wishes, –jr

John H. Rex, MD | Chief Medical Officer, F2G Ltd. | Expert-in-Residence, Wellcome Trust. Follow me on Twitter: @JohnRex_NewAbx. See past newsletters and subscribe for the future: https://13.43.35.2/blog/

Upcoming meetings of interest to the AMR community:

• 10 June 2019 (everywhere): Application deadline for NIAID solicitation (HHS-NIH-NIAID-BAA2019-1) for proposals to support new vaccine or therapeutics candidates targeting antibiotic-resistant bacterial infections. Go here for more details.
• 3-6 Jun 2019 (Philadelphia): Annual BIO meeting
• 4 Jun 2019 (webinar, 4 June, 13:30-15:00 CEST / 17:00-18:30 IST / 18:30-20:00 ICT). Repeat of REVIVE webinar entitled “Mining Chemical Libraries for New Antibacterials”. Go here to register.
• 11 June 2019 (webinar, 17:00-18:30 CEST): REVIVE webinar entitled “Models for antimicrobial R&D: Development and use of in vivo models for infectious disease research”. Go here to register
• 11 June 2019 (webinar, 11.00-12.00 EST): CARB-X and CFF (Cystic Fibrosis Foundation) webinar on CFF’s new infection research initiative. Go here to register.
• 20-24 June 2019 (San Francisco): Annual ASM Microbe meeting.
• 9 July 2019 (webinar, 17:00-18:30 CEST): REVIVE webinar entitled “Infection models for antimicrobial R&D: Intracellular models”. Go here to register.
• 10-11 Jul 2019 (Madison, WI): Tiny Earth Symposium, a teaching consortium that uses crowd-sourcing of antibiotic-producing microbes to improve undergraduate education. Details here.
• [NEW] 11-12 July 2019 (Washington, DC): FDA-sponsored workshop entitled “Leveraging Randomized Clinical Trials to Generate Real-World Evidence for Regulatory Purposes.” Go here for more details.
• 12 July 2019 (FDA, White Oak Campus): Public workshop to discuss the 2018 LPAD guidance. Register here.
• [Mark your calendar now!] 3-6 Sep 2019 (Boston). Annual ASM-ESCMID Conference on Antibiotic Development. The Bootcamp series will continue on 3 Sep with main meeting on 4-6 Sep. Mark your calendar now and check back here for details.
• 6-8 Sep 2019 (Bilbao, Spain): 5th ESCMID conference on Vaccines. Check back here for details.
• 2-6 Oct 2019 (Washington, DC): IDSA’s annual IDWeek meeting.
• 19-27 Oct 2019 (Annecy, France): International Course on Antibiotics and Resistance (ICARe) – A soup-to-nuts intensive residential training program on all things AMR, especially R&D for new antibiotics. See this link for details.
• 1-6 Mar 2020 (Il Ciocco, Tuscany, Italy): GRC on Antibacterial Discovery and Development: “Now is the time to re-boot antibiotic R&D before it’s too little, too late.” Go here for details.
• 12-13 Mar 2020 (Berlin?): BEAM-, Novo REPAIR-, CARB-X-, DZIF-, ND4BB-, ENABLE-supported (among a long list!) Conference on Novel Antimicrobials and AMR Diagnostics. Final location is TBD, details will appear here, and you should mark your calendar now. 
• 16-17 Mar 2020 (London): BSAC Spring Conference entitled: “Bridging the gap between science, policy and effective antimicrobial use.” Go here for details. 
• 18-21 Apr 2020 (Paris): Annual ECCMID meeting (#30)
• 10-13 Apr 2021 (Vienna): Annual ECCMID meeting (#31)